What we know about the therapeutic landscape of desmoid tumors: an analysis of clinical trials registered on clinicaltrials.gov

Summary

This review analyzes the evolving therapeutic landscape of desmoid tumors (DTs) through a comprehensive evaluation of clinical trials registered on ClinicalTrials.gov from 1978 to 2025. A total of 62 DT-related studies were identified, including 23 completed interventional trials investigating systemic and locoregional therapies. Most studies focused on treatment efficacy and safety, with increasing interest in targeted therapies such as tyrosine kinase inhibitors, γ-secretase inhibitors, Wnt/β-catenin pathway inhibitors, and mTOR-targeted agents. Among the most promising therapies, nirogacestat, pazopanib, sorafenib, and imatinib demonstrated meaningful clinical activity and favorable progression-free survival outcomes in patients with progressive or unresectable disease. Locoregional approaches, including radiotherapy and cryoablation, also showed beneficial tumor control and symptom relief. Despite the expanding therapeutic armamentarium, only a small proportion of trials reported published results, highlighting a persistent knowledge gap in DT management. The study emphasizes the need for individualized, multidisciplinary treatment strategies and further molecularly driven research to optimize outcomes for patients with desmoid tumors.